Myelodysplastic SyndromePhase 3SINTRA-REVNCT01243476
Study of REVLIMID (Lenalidomide) Versus Placebo in Patients With Low Risk Myelodysplastic Syndrome
A Phase 3 study testing Lenalidomide in people with Myelodysplastic Syndrome, run by Fundación General de la Universidad de Salamanca at 25 sites in 3 countries.
Trial summary
Trial Design: This clinical trial is a phase III multicenter, randomized, double blind and controlled with placebo trial and with two arms designed to assess the efficiency and toxicity of the scheme Lenalidomide versus observation in a series of 60 patients with low risk myelodysplastic syndrome associated to 5q… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- Lenalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 61 participants
- Where
- 25 sitesin 3 countries
- Study dates
- Jan 2010to 6 Jun 2022
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- - The patient must, in the investigator's opinion, be able to comply with all the clinical trial requirements.
- - The patient must voluntarily sign the informed consent form before undergoing any test of the trial that is not part of the normal patient care, and patient must be aware that he/she can withdraw from the trial at any time, without it ever affecting their future healthcare.
- - Age > 18 years.
- - The patient must be diagnosed with low risk MDS (low and intermediate-1 IPSS) associated with 5q deletion, either as an isolated abnormality or accompanied by other additional cytogenetic abnormalities.
- - MDS Del(5q) with transfusion-independent anaemia (Hb ≤ 12 g/dL), and documented confirmation that no packed red blood cells transfusion due to the patient's underlying condition (MDS) has been received.
- - The patient must have an ECOG performance status of ≤ 2.
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- - The patient must be able to comply with the scheduled study visits.
- - Female patient with childbearing potential must*:
- Understands the teratogenic risk of the study drug.
- Commits herself to use two forms of effective birth control continuously, and is able to use them correctly, for the 4 weeks prior to starting treatment with the study drug, as well as during treatment with the study drug (including periods of dose interruption), and for up to 4 weeks after finishing treatment with the study drug, even if amenorrhoeic. This always applies, except in women who commit to continued complete sexual abstinence, as confirmed on a monthly basis.
- The patient must understand that even if she is amenorrhoeic she must follow all the advice on effective contraception.
- The patient must understand the possible consequences of pregnancy and the need to attend a healthcare service urgently in case there is a risk of pregnancy.
- Agree to undergo a pregnancy test with a minimum sensitivity of 25 mIU/mL, under medical supervision, on the day of the study visit or during the 3 days prior to this visit, after using effective birth control for at least 4 weeks. This requirement also applies to women with childbearing potential who practice complete and continued sexual abstinence. The test must confirm that the patient is not pregnant at the time the treatment is initiated.
- Agree to undergo a pregnancy test, under medical supervision, weekly for he first 28 days of treatment, and subsequently every 4 weeks, including a pregnancy test 4 weeks after finishing the study treatment, except in case of confirmed tubal ligation. This pregnancy test will be performed on the day of the study visit or during the 3 days prior to it. This requirement also applies to women with childbearing potential who practice complete and continued sexual abstinence.
- - All male patients must:
- Commit himself to the use of condoms throughout all the treatment with the study drug, including all periods of dose interruption, and up to one week after finishing the treatment if their partner is a woman with childbearing potential and does not use birth control methods.
- Commit himself to not donate semen during treatment with the study drug and up to one week after finishing the treatment.
- - All patients must:
- Refrain from donating blood while receiving treatment with the study drug and during the week following the end of the treatment.
- Refrain from sharing the study drug with others, and return all unused study drug to the investigator or pharmacist.
Who cannot join
- - Any organic disease or psychiatric disorder which makes it impossible for the patient to sign or understand the informed consent.
- - Having received any treatment for MDS.
- - Del(5q) MDS with transfusion-dependent anaemia, and documented confirmation that the patient has received any pRBC transfusion due to the underlying condition (MDS).
- - Pregnant or breast-feeding women.
- - Any of the following laboratory abnormalities:
- Absolute neutrophil count < 500/mm3
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- Platelet count < 25,000/mm3
- Serum GOT or GPT > 3 times the upper limit of normal values.
- Total serum bilirubin > 2 times the upper limit of normal values.
- - Previous history of other malignancies other than MDS (except for basal cell or squamous cell skin carcinoma, or carcinoma in situ of the cervix or breast), unless the patient has been free of disease for more than 5 years.
- - Known hypersensitivity to or a history of uncontrollable side effects to lenalidomide.
- - Major surgery within the 4 weeks prior to the inclusion in the trial.
- - The patient has received any investigational agent in the 30 days prior to inclusion.
Trial locations and contacts
25 sites in 3 countries.
25 sites in 3 countries
Spain15 sites
Hospital Universitari Germans Trias i Pujol (ICO Badalona)
Badalona, Barcelona, 08916
Hospital de Cruces
Barakaldo, Vizcaya, 48930
Hospital Clínic i Provincial
Barcelona, 08036
Hospital Universitario Reina Sofía
Córdoba, 14004
Hospital de Cabueñes
Gijón, Principality of Asturias, 33394
Instituto Catalán de Oncología de Gerona
Girona, 17007
Hospital Infanta Leonor
Madrid, 28031
Hospital Clínico San Carlos
Madrid, 28040
Hospital Universitario La Paz
Madrid, 28046
Hospital General Universitario José Maria Morales Meseguer
Murcia, 30008
Hospital Central de Asturias
Oviedo, Principality of Asturias, 33006
Hospital Son Llàtzer
Palma de Mallorca, Balearic Islands, 07198
Hospital Clínico Universitario de Salamanca
Salamanca, 37007
Hospital Universitario Virgen del Rocío
Seville, 41013
Hospital Universitario La Fe
Valencia, 46009
France7 sites
Centre Hospitalier Universitaire d'Angers
Angers, Maine Y Loira, 49100
Centre Hospitalier d'Avignon
Avignon, Vaucluse, 84000
Centre Hospitalier Universitaire Brabois
Nancy, Meurthe Y Mosel, 54500
Centre Hospitalier Universitaire de Nîmes
Nîmes, Gard, 30900
Centre Hospitalier Régional d'Orléans
Orléans, Loiret, 45100
Hôspital St. Louis
Paris, 75010
Hôpitaux Universitaires de Strasbourg
Strasbourg, Bajo Rin, 67091
Germany3 sites
Universitaetsklinikum Carl Gustav Carus der Technischen Universitaet Dresden
Dresden, 01307
Marien Hospital Duesseldorf
Düsseldorf, 40479
Klinikum rechts der Isar der Technischen Universität München
München, 81675
About this study
Official title: Multicenter, Randomized, Double-blind, Phase III Study of REVLIMID (Lenalidomide) Versus Placebo in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) With Alteration in 5q- and Anemia Without the Need of Transfusion.
Trial Design:
This clinical trial is a phase III multicenter, randomized, double blind and controlled with placebo trial and with two arms designed to assess the efficiency and toxicity of the scheme Lenalidomide versus observation in a series of 60 patients with low risk myelodysplastic syndrome associated to 5q deletion with anemia (Hb≤12g/dL) but without the need of transfusion. Patients are randomized in the study in a 2:1 ratio. They will receive treatment for 104 weeks until progression of the disease, which implies that the patient suffering from anemia due to myelodysplastic syndrome requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months), or unacceptable toxicity.
Read more
Disease:
Low risk myelodysplastic syndrome associated to the loss of 5q without transfusion requirements.
Total number of patients:
In total 60 patients will be included, 40 assigned to the treatment branch and 20 to the placebo branch.
Calendar:
First patient first visit: February 2010, and Last patient last visit expected in February 2016. (Recruitment was initially expected to take place over a period of 24 months and was expected to be finished in February 2012, but due to low rate of recruitment it was extended until the population sample is included in the trial).
Read the detailed description
General summary of myelodysplastic syndrome with alteration in 5q. Myelodysplastic syndromes (MDS) are a very heterogenic group of diseases characterized by the presence of morphologic features of dyshemopoiesis in bone marrow (BM) and in peripheral blood (PB), which is translated into an inefficient hematopoiesis. This will lead to the concomitant development of peripheral cytopenias which will be the cause of complications in these patients and, in most cases, the cause of death. In addition, these patients have an increased risk of developing acute leukemia, and this risk increases with the progression of the disease.MDS represents an incurable disease with standard treatments with a quite variable survival mean ranging from 3 months to 15 years depending on the number of blasts, the number of cytopenias and the type of cytogenetic disorders2. The sole curative treatment of the MDS is the allogenic transplant of hematopoietic progenitors but this option is only available for a 5% of the patients with MDS.
Lenalidomide, initially known as CC-5013, is an analogue of the immunomodulator Drug Thalidomide (Thalidomid®; Pharmion Corp., Boulder, CO, USA), which was the first drug with anti-angiogenic and immunomodulator properties investigated in MDS. Lenalidomide has, as well as thalidomide, a broad array of potential activities against dysplastic cells, not fully known, among which we find immunomodulator and non-immunomodulator properties (anti-angiogenic effect, anti-proliferative and pro-apoptotic). The greater advantage of Lenalidomide in comparison with thalidomide is that the former is between 50 and 2000 times stronger tan thalidomide in what respects to its immunomodulator effects. And moreover, the toxic profile of Lenalidomide seems lower than that of its analogue; Thalidomide.
There is no treatment indication on the present times for patients with low risk MDS associated to the loss of 5q without transfusion dependent anemia. The results with Lenalidomide are highly promising for patients with low risk MDS associated to the loss of 5q when (it has so been tested) there is red blood cells transfusion dependent anemia. In this sense, the proposal consists on being able to state that treatment with Lenalidomide can be efficient from diagnose preventing CH transfusions with an acceptable toxicity.
In this sense, the present study has the intention to treat early patients with low-risk MDS associated to the loss of 5q with a view to prevent CH transfusion. Therefore, we could extend in these patients the time until CH transfusion and even assess the possibility of eradicating the disease at a cytogenetic/morphologic level. In the present trial and given the characteristics of the patients, we have chosen the option of supplying Lenalidomide at a dose of 5mg/day. The option of considering a lower dose to the one currently considered as "standard" (10mg/day) is to reduce toxicity, mainly hematologic, in patients who do not receive treatment normally. A dose with lower toxicity has been chosen which has revealed itself efficient.
Main efficiency objective:
•To assess if treatment with Revlimid (Lenalidomide) extends the period until the progression to myelodysplastic syndrome del(5q) considered as transfusion independent, documented verification that the patient suffering from anemia due to MDS requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months). Revlimid will be compared to the current standard treatment for patients with low risk myelodysplastic syndrome associated with the loss of 5qwithout transfusion dependent anemia, which is the therapeutic abstention and monitoring until its progression.
Secondary efficiency objectives:
- Erythroid response according to the Criteria of the myelodysplastic syndrome International Work Team 2006).
- Duration of the red blood cells transfusion independency (defined as the number of days elapsed between the randomization and the first transfusion after this period free of transfusions).
- Change of hemoglobin concentration (Hb) in relation to baseline levels of patients who show erythroid response.
- Variation in platelets absolute count in relation to baseline levels.
- Variation in neutrophils absolute count in relation to baseline levels.
- Cytogenetic response according to the Criteria of the myelodysplastic syndrome International Work Team.
- Bone marrow response according to the Criteria of the myelodysplastic syndrome International Work Team.
- To assess the safety and tolerance of the Lenalidomide scheme, measured according to the incidence of clinical and laboratory toxicity.
- Global survival, Event Free Survival and Rate of Transformation to Acute Leukemia.
- Time from diagnose to transfusion independence.
Main safety objective:
Safety (type, frequency and severity [Criteria of normal terminology of adverse reactions of the National Cancer Institute (NCI CTCAE) version 3.0] of adverse reactions (AR)and list of the AR with Lenalidomide.
Four of the sites of this Clinical Trial are member of the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet) which is partially co-funded by the European Union within the framework of the Third Health Programme. "ERN-2016 - Framework Partnership Agreement 2017-2021." FPA 739541".
- Conditions
- Myelodysplastic Syndrome
- Being tested
- Lenalidomide, Placebo
- Keywords
- myelodysplastic syndrome, 5q deletion, anemia, lenalidomide, red blood cells transfusion
- Medical subject headings
- Myelodysplastic Syndromes, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LenalidomideDrug
Also called Revlimid 5 mg
Treatment with Revlimid (lenalidomide), oral use, 5 mg daily during study treatment (104 weeks).
PlaceboOther
Placebo, oral use, daily during study treatment (104 weeks)
| Study group | Role | What they receive |
|---|---|---|
| lenalidomide | Experimental | Experimental treatment branch with Lenalidomide 5 mg/day (oral use) Receives: Drug: Lenalidomide |
| placebo | Placebo comparator | Placebo branch (oral use) Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Period until the progression of myelodysplastic syndrome
To assess if treatment with Revlimid (Lenalidomide) extends the period until the progression to MDS of(5q) considered as transfusion independent, documented verification that the patient suffering from anemia due to MDS requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months). Revlimid will be compared to the current standard treatment for patients with low risk MDS associated with the loss of 5q without transfusion dependent anemia, which is the therapeutic abstention and monitoring until its progression.
Measured: 6 years (study treatment and follow up)
Secondary outcomes (9)
Erythroid response
Erythroid response according to the Criteria of the MDS International Work Team.
Measured: 6 years (study treatment and follow up)
Duration of the red blood cells transfusion independency
Red blood cells transfusion independency,defined as the number of days elapsed between the randomization and the first transfusion after this period free of transfusions.
Measured: 6 years (study treatment and follow up)
Change of the hemoglobin concentration (Hb) in relation to baseline levels
Change of the hemoglobin concentration (Hb) in relation to baseline levels of patients who show erythroid response.
Measured: 6 years (study treatment and follow up)
Variation in platelets and neutrophils absolute count in relation to baseline levels
Variation in platelets and neutrophils absolute count in relation to baseline levels
Measured: 6 years (study treatment and follow up)
Cytogenetic response
Cytogenetic response according to the Criteria of the MDS International Work Team.
Measured: 6 years (study treatment and follow up)
Bone marrow response
Bone marrow response according to the Criteria of the MDS International Work Team.
Measured: 6 years (study treatment and follow up)
Global survival, Event Free Survival and Rate of Transformation to Acute Leukemia.
Global survival, Event Free Survival and Rate of Transformation to Acute Leukemia
Measured: 6 years (study treatment and follow up)
Time from diagnose to transfusion independence.
Time from diagnose to transfusion independence
Measured: 6 years (study treatment and follow up)
Safety( type, frequency and severity of adverse events and relationship to lenalidomide
Safety (type, frequency and severity [Criteria of normal terminology of adverse reactions of the National Cancer Institute (NCI CTCAE) version 3.0] of adverse reactions (AR)and list of the AR with Lenalidomide.
Measured: 6 years (study treatment and follow up)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2010 (estimated)
- Study startJan 2010
- Primary completion6 Jun 2022
- Study completion6 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundación General de la Universidad de Salamanca
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- Consuelo del Cañizo, MD, Study chair, Hospital Clínico Universitario de SalamancaMaría Díez Campelo, MD, Study chair, Hospital Clínico Universitario de Salamanca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01243476
- Sponsor's protocol ID
- SINTRA-REV
- Other IDs
- 2009-013619-36, 2009
- Acronym
- SINTRA-REV
- Status last verified
- Jan 2023
- First submitted
- 17 Nov 2010
- Last updated
- 15 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
Publications
Steensma DP, Tefferi A. The myelodysplastic syndrome(s): a perspective and review highlighting current controversies. Leuk Res. 2003 Feb;27(2):95-120. doi: 10.1016/s0145-2126(02)00098-x.
PubMed 12526916Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997 Mar 15;89(6):2079-88.
PubMed 9058730List AF. New approaches to the treatment of myelodysplasia. Oncologist. 2002;7 Suppl 1:39-49. doi: 10.1634/theoncologist.7-suppl_1-39.
PubMed 11961208McHugh SM, Deighton J, Stewart AG, Lachmann PJ, Ewan PW. Bee venom immunotherapy induces a shift in cytokine responses from a TH-2 to a TH-1 dominant pattern: comparison of rush and conventional immunotherapy. Clin Exp Allergy. 1995 Sep;25(9):828-38. doi: 10.1111/j.1365-2222.1995.tb00025.x.
PubMed 8564721Bellamy WT. Expression of vascular endothelial growth factor and its receptors in multiple myeloma and other hematopoietic malignancies. Semin Oncol. 2001 Dec;28(6):551-9. doi: 10.1016/s0093-7754(01)90023-5.
PubMed 11740808Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents. Nat Rev Cancer. 2004 Apr;4(4):314-22. doi: 10.1038/nrc1323. No abstract available.
PubMed 15057291List A, Kurtin S, Roe DJ, Buresh A, Mahadevan D, Fuchs D, Rimsza L, Heaton R, Knight R, Zeldis JB. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005 Feb 10;352(6):549-57. doi: 10.1056/NEJMoa041668.
PubMed 15703420List A, Dewald G, Bennett J, Giagounidis A, Raza A, Feldman E, Powell B, Greenberg P, Thomas D, Stone R, Reeder C, Wride K, Patin J, Schmidt M, Zeldis J, Knight R; Myelodysplastic Syndrome-003 Study Investigators. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5;355(14):1456-65. doi: 10.1056/NEJMoa061292.
PubMed 17021321List A, Gordon W, Dewald G, Bennett J, Giagounidis A, Raza A et al. Long-term clinical benefit of lenalidomide (Revlimid) treatment in patients with myelodysplastic syndrome and chromosome deletion 5q [Abstract]. Blood. 2006;108:251A.
Mallo M, Cervera J et al. Prognostic impact of additional chromosomal aberrations to 5q- in patients with primary myelodysplastic syndromes. Oral comunication, 0906, 13th Congress of the European Hematology Association. Haematologica | 2008; 93(s1), pag. 360
Fenaux P, Kelaidi C. Treatment of the 5q- syndrome. Hematology Am Soc Hematol Educ Program. 2006:192-8. doi: 10.1182/asheducation-2006.1.192.
PubMed 17124060Kelaidi C, Eclache V, Fenaux P. The role of lenalidomide in the management of myelodysplasia with del 5q. Br J Haematol. 2008 Feb;140(3):267-78. doi: 10.1111/j.1365-2141.2007.06910.x.
PubMed 18217896List, AF. Active treatment-improving outcomes in del 5q patients. Leukemia Research, (2007)31(Suppl. 1), S9.
Diez-Campelo M, Lopez-Cadenas F, Xicoy B, Lumbreras E, Gonzalez T, Del Rey Gonzalez M, Sanchez-Garcia J, Coll Jorda R, Slama B, Hernandez-Rivas JA, Thepot S, Bernal T, Guerci-Bresler A, Bargay J, Amigo ML, Preudhomme C, Fenwarth L, Platzbecker U, Gotze KS, Arar A, Toribio S, Del Canizo C, Hernandez-Rivas JM, Fenaux P. Low dose lenalidomide versus placebo in non-transfusion dependent patients with low risk, del(5q) myelodysplastic syndromes (SintraREV): a randomised, double-blind, phase 3 trial. Lancet Haematol. 2024 Sep;11(9):e659-e670. doi: 10.1016/S2352-3026(24)00142-X. Epub 2024 Jul 18.
PubMed 39033767
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomized, Double-blind, Phase III Study of REVLIMID (Lenalidomide) Versus Placebo in Patients With Low Risk Myelodysplastic Syndrome (Low and Intermediate-1 IPSS) With Alteration in 5q- and Anemia Without the Need of Transfusion.
- ClinicalTrials.gov ID
- NCT01243476
- Acronym / sponsor ID
- SINTRA-REV / SINTRA-REV
- Conditions
- Myelodysplastic Syndrome
- Keywords
- myelodysplastic syndrome, 5q deletion, anemia, lenalidomide, red blood cells transfusion
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Fundación General de la Universidad de SalamancaUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- Jan 2010
- Primary completion
- 6 Jun 2022 (actual)
- Study completion
- 6 Jun 2022 (actual)
- First posted
- 18 Nov 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Trial Design:
This clinical trial is a phase III multicenter, randomized, double blind and controlled with placebo trial and with two arms designed to assess the efficiency and toxicity of the scheme Lenalidomide versus observation in a series of 60 patients with low risk myelodysplastic syndrome associated to 5q deletion with anemia (Hb≤12g/dL) but without the need of transfusion. Patients are randomized in the study in a 2:1 ratio. They will receive treatment for 104 weeks until progression of the disease, which implies that the patient suffering from anemia due to myelodysplastic syndrome requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months), or unacceptable toxicity.
Disease:
Low risk myelodysplastic syndrome associated to the loss of 5q without transfusion requirements.
Total number of patients:
In total 60 patients will be included, 40 assigned to the treatment branch and 20 to the placebo branch.
Calendar:
First patient first visit: February 2010, and Last patient last visit expected in February 2016. (Recruitment was initially expected to take place over a period of 24 months and was expected to be finished in February 2012, but due to low rate of recruitment it was extended until the population sample is included in the trial).
Detailed description
General summary of myelodysplastic syndrome with alteration in 5q. Myelodysplastic syndromes (MDS) are a very heterogenic group of diseases characterized by the presence of morphologic features of dyshemopoiesis in bone marrow (BM) and in peripheral blood (PB), which is translated into an inefficient hematopoiesis. This will lead to the concomitant development of peripheral cytopenias which will be the cause of complications in these patients and, in most cases, the cause of death. In addition, these patients have an increased risk of developing acute leukemia, and this risk increases with the progression of the disease.MDS represents an incurable disease with standard treatments with a quite variable survival mean ranging from 3 months to 15 years depending on the number of blasts, the number of cytopenias and the type of cytogenetic disorders2. The sole curative treatment of the MDS is the allogenic transplant of hematopoietic progenitors but this option is only available for a 5% of the patients with MDS.
Lenalidomide, initially known as CC-5013, is an analogue of the immunomodulator Drug Thalidomide (Thalidomid®; Pharmion Corp., Boulder, CO, USA), which was the first drug with anti-angiogenic and immunomodulator properties investigated in MDS. Lenalidomide has, as well as thalidomide, a broad array of potential activities against dysplastic cells, not fully known, among which we find immunomodulator and non-immunomodulator properties (anti-angiogenic effect, anti-proliferative and pro-apoptotic). The greater advantage of Lenalidomide in comparison with thalidomide is that the former is between 50 and 2000 times stronger tan thalidomide in what respects to its immunomodulator effects. And moreover, the toxic profile of Lenalidomide seems lower than that of its analogue; Thalidomide.
There is no treatment indication on the present times for patients with low risk MDS associated to the loss of 5q without transfusion dependent anemia. The results with Lenalidomide are highly promising for patients with low risk MDS associated to the loss of 5q when (it has so been tested) there is red blood cells transfusion dependent anemia. In this sense, the proposal consists on being able to state that treatment with Lenalidomide can be efficient from diagnose preventing CH transfusions with an acceptable toxicity.
In this sense, the present study has the intention to treat early patients with low-risk MDS associated to the loss of 5q with a view to prevent CH transfusion. Therefore, we could extend in these patients the time until CH transfusion and even assess the possibility of eradicating the disease at a cytogenetic/morphologic level. In the present trial and given the characteristics of the patients, we have chosen the option of supplying Lenalidomide at a dose of 5mg/day. The option of considering a lower dose to the one currently considered as "standard" (10mg/day) is to reduce toxicity, mainly hematologic, in patients who do not receive treatment normally. A dose with lower toxicity has been chosen which has revealed itself efficient.
Main efficiency objective:
•To assess if treatment with Revlimid (Lenalidomide) extends the period until the progression to myelodysplastic syndrome del(5q) considered as transfusion independent, documented verification that the patient suffering from anemia due to MDS requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months). Revlimid will be compared to the current standard treatment for patients with low risk myelodysplastic syndrome associated with the loss of 5qwithout transfusion dependent anemia, which is the therapeutic abstention and monitoring until its progression.
Secondary efficiency objectives:
- Erythroid response according to the Criteria of the myelodysplastic syndrome International Work Team 2006).
- Duration of the red blood cells transfusion independency (defined as the number of days elapsed between the randomization and the first transfusion after this period free of transfusions).
- Change of hemoglobin concentration (Hb) in relation to baseline levels of patients who show erythroid response.
- Variation in platelets absolute count in relation to baseline levels.
- Variation in neutrophils absolute count in relation to baseline levels.
- Cytogenetic response according to the Criteria of the myelodysplastic syndrome International Work Team.
- Bone marrow response according to the Criteria of the myelodysplastic syndrome International Work Team.
- To assess the safety and tolerance of the Lenalidomide scheme, measured according to the incidence of clinical and laboratory toxicity.
- Global survival, Event Free Survival and Rate of Transformation to Acute Leukemia.
- Time from diagnose to transfusion independence.
Main safety objective:
Safety (type, frequency and severity [Criteria of normal terminology of adverse reactions of the National Cancer Institute (NCI CTCAE) version 3.0] of adverse reactions (AR)and list of the AR with Lenalidomide.
Four of the sites of this Clinical Trial are member of the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet) which is partially co-funded by the European Union within the framework of the Third Health Programme. "ERN-2016 - Framework Partnership Agreement 2017-2021." FPA 739541".
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| lenalidomide | Experimental | Drug: LenalidomideExperimental treatment branch with Lenalidomide 5 mg/day (oral use) |
| placebo | Placebo comparator | Other: PlaceboPlacebo branch (oral use) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- - The patient must, in the investigator's opinion, be able to comply with all the clinical trial requirements.
- - The patient must voluntarily sign the informed consent form before undergoing any test of the trial that is not part of the normal patient care, and patient must be aware that he/she can withdraw from the trial at any time, without it ever affecting their future healthcare.
- - Age > 18 years.
- - The patient must be diagnosed with low risk MDS (low and intermediate-1 IPSS) associated with 5q deletion, either as an isolated abnormality or accompanied by other additional cytogenetic abnormalities.
- - MDS Del(5q) with transfusion-independent anaemia (Hb ≤ 12 g/dL), and documented confirmation that no packed red blood cells transfusion due to the patient's underlying condition (MDS) has been received.
Key exclusion criteria (13 listed)
- - Any organic disease or psychiatric disorder which makes it impossible for the patient to sign or understand the informed consent.
- - Having received any treatment for MDS.
- - Del(5q) MDS with transfusion-dependent anaemia, and documented confirmation that the patient has received any pRBC transfusion due to the underlying condition (MDS).
- - Pregnant or breast-feeding women.
- - Any of the following laboratory abnormalities:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LenalidomideAlso: Revlimid 5 mgTreatment with Revlimid (lenalidomide), oral use, 5 mg daily during study treatment (104 weeks). | Drug | lenalidomide |
| PlaceboPlacebo, oral use, daily during study treatment (104 weeks) | Other | placebo |
Primary outcome
Period until the progression of myelodysplastic syndrome
Time frame: 6 years (study treatment and follow up)
To assess if treatment with Revlimid (Lenalidomide) extends the period until the progression to MDS of(5q) considered as transfusion independent, documented verification that the patient suffering from anemia due to MDS requires transfusion of at least 2 UCH/56 days (2 months) with a minimum follow up of 112 days (4 months). Revlimid will be compared to the current standard treatment for patients with low risk MDS associated with the loss of 5q without transfusion dependent anemia, which is the therapeutic abstention and monitoring until its progression.
9 secondary outcomes
Erythroid response
Time frame: 6 years (study treatment and follow up)
Duration of the red blood cells transfusion independency
Time frame: 6 years (study treatment and follow up)
Change of the hemoglobin concentration (Hb) in relation to baseline levels
Time frame: 6 years (study treatment and follow up)
Variation in platelets and neutrophils absolute count in relation to baseline levels
Time frame: 6 years (study treatment and follow up)
Cytogenetic response
Time frame: 6 years (study treatment and follow up)
Bone marrow response
Time frame: 6 years (study treatment and follow up)
Global survival, Event Free Survival and Rate of Transformation to Acute Leukemia.
Time frame: 6 years (study treatment and follow up)
Time from diagnose to transfusion independence.
Time frame: 6 years (study treatment and follow up)
Safety( type, frequency and severity of adverse events and relationship to lenalidomide
Time frame: 6 years (study treatment and follow up)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2023.
- Background: PubMed 12526916Steensma DP, Tefferi A. The myelodysplastic syndrome(s): a perspective and review highlighting current controversies. Leuk Res. 2003 Feb;27(2):95-120. doi: 10.1016/s0145-2126(02)00098-x.
- Background: PubMed 9058730Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, Sanz M, Vallespi T, Hamblin T, Oscier D, Ohyashiki K, Toyama K, Aul C, Mufti G, Bennett J. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997 Mar 15;89(6):2079-88.
- Background: PubMed 11961208List AF. New approaches to the treatment of myelodysplasia. Oncologist. 2002;7 Suppl 1:39-49. doi: 10.1634/theoncologist.7-suppl_1-39.
- Background: PubMed 8564721McHugh SM, Deighton J, Stewart AG, Lachmann PJ, Ewan PW. Bee venom immunotherapy induces a shift in cytokine responses from a TH-2 to a TH-1 dominant pattern: comparison of rush and conventional immunotherapy. Clin Exp Allergy. 1995 Sep;25(9):828-38. doi: 10.1111/j.1365-2222.1995.tb00025.x.
- Background: PubMed 11740808Bellamy WT. Expression of vascular endothelial growth factor and its receptors in multiple myeloma and other hematopoietic malignancies. Semin Oncol. 2001 Dec;28(6):551-9. doi: 10.1016/s0093-7754(01)90023-5.
- Background: PubMed 15057291Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents. Nat Rev Cancer. 2004 Apr;4(4):314-22. doi: 10.1038/nrc1323. No abstract available.
- Background: PubMed 15703420List A, Kurtin S, Roe DJ, Buresh A, Mahadevan D, Fuchs D, Rimsza L, Heaton R, Knight R, Zeldis JB. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005 Feb 10;352(6):549-57. doi: 10.1056/NEJMoa041668.
- Background: PubMed 17021321List A, Dewald G, Bennett J, Giagounidis A, Raza A, Feldman E, Powell B, Greenberg P, Thomas D, Stone R, Reeder C, Wride K, Patin J, Schmidt M, Zeldis J, Knight R; Myelodysplastic Syndrome-003 Study Investigators. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5;355(14):1456-65. doi: 10.1056/NEJMoa061292.
- BackgroundList A, Gordon W, Dewald G, Bennett J, Giagounidis A, Raza A et al. Long-term clinical benefit of lenalidomide (Revlimid) treatment in patients with myelodysplastic syndrome and chromosome deletion 5q [Abstract]. Blood. 2006;108:251A.
- BackgroundMallo M, Cervera J et al. Prognostic impact of additional chromosomal aberrations to 5q- in patients with primary myelodysplastic syndromes. Oral comunication, 0906, 13th Congress of the European Hematology Association. Haematologica | 2008; 93(s1), pag. 360
- Background: PubMed 17124060Fenaux P, Kelaidi C. Treatment of the 5q- syndrome. Hematology Am Soc Hematol Educ Program. 2006:192-8. doi: 10.1182/asheducation-2006.1.192.
- Background: PubMed 18217896Kelaidi C, Eclache V, Fenaux P. The role of lenalidomide in the management of myelodysplasia with del 5q. Br J Haematol. 2008 Feb;140(3):267-78. doi: 10.1111/j.1365-2141.2007.06910.x.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Consuelo del Cañizo, MD study chair
Hospital Clínico Universitario de Salamanca
María Díez Campelo, MD study chair
Hospital Clínico Universitario de Salamanca
25 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lenalidomide in people with Myelodysplastic Syndrome, run by Fundación General de la Universidad de Salamanca at 25 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in Spain, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Period until the progression of myelodysplastic syndrome (time frame: 6 years (study treatment and follow up)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01243476, which also lists 14 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Feb 2023.